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PHARMA: Where are the orphan cancer drugs? by The Industry Veteran

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Greg Pawelski’s recent posting here about whether cancer care could improve has drawn several responses, many of which I’m trying to redirect back into the comments section.  One, however,  that deserves its own posting is from our old friend The Industry Veteran who wonders whether the current model of big pharma itself can sustain itself in the kind kind of designer-drug world Greg is proposing:

Greg Pawelski makes a good point in his post, specifically, that the economic
model under which Pharma companies develop oncology products may be inimical to
the individualized treatment approaches required for the disease(s).  I suspect
that the figurative lumber rooms of the Big Pharma companies are stacked with
discontinued oncology compounds that proved wonderfully effective and tolerable
for 5%-10% of the target candidates but, sadly, they were terribly toxic or no
better than placebo for the others.  In fact, I know that’s the case.  Not long
ago I completed a study for a client to find why the companies are so reluctant
to out-license these moribund compounds.  The overwhelming answer, the one that
dwarfs all the others, is ego of the fiduciary executives.  The executives feel
they would invite serious job trouble by out-licensing an abandoned compound to
a small startup that proceeds to make it a successful brand.
While
Pawelski deplores the one-size-fits-all requirement, that constitutes the
standard among Big Pharma for launching a product. 

Today the Big Pharmas will
curtail development of a product with projected, peak year, global sales of less
than $850 million.  Anything less will not sustain their high fixed costs or
permit the economies of scale on variable expenses that represents their
comparative advantage.  When BusinessWeek asked Pfizer’s CEO Hank
McKinnell if the era of the blockbuster (and the giant Pharmas created to
support such megaliths) has passed, he replied, “Anybody who says that doesn’t
understand our business.”  A small company, however, can derive a large return
on equity/sales/assets from a product that successfully treats 5%-10% of a
comparatively small, target population.  The CEO of such a company probably
won’t receive $50 million annual compensation the way Mr. McKinnell does, but
its stockholders and a reasonable number of patients can benefit when a Celgene
takes an abandoned and despised compound (thalidomide, developed as a
tranquilizer for pregnant women) and brings it to market as a major therapy for
multiple myeloma.

I suspect this dilemma will resolve itself as the
pharmaceutical industry evolves into what Oracle’s CEO, Larry Ellison, once
called the Hollywood approach to drug development.  I’ve written about this
before and don’t wish to repeat myself, but basically this involves the Big
Pharmas limiting themselves to acting as sources for development funding and
distribution, while independent producers (biotechs? specialty companies?) buy
the properties and develop them.  Movies today can successfully reach smaller,
more segmented audiences than the big Hollywood studios ever could during the
Mickey Rooney, Judy Garland days.  Unfortunately a lot of people will needlessly
die of cancer before Andy Hardy grows up and tells Louis B. Mayer to go screw
himself.  But hey, in a country that twice elects a wannabe redneck as
president, the market is sacrosanct and its pace of Darwinian change is all we
can expect.

POLICY: The high cost of health care

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I’m giving a talk this morning about consumer health care so not much time for a long post.  Kind of ironic that we’re entering the brave new world of consumer health care at a time when the price of insurance has got so high many people just cannot afford it. The LA Times today has an article called At what cost? which has some horrendous stories of low paid workers having to pay up to a quarter of their income in health insurance premiums. As IFTF/Harris has said for years, the empowered consumer is in general reluctantly empowered, and the basic ethos is pay more, get less.

POLICY/POLITICS: Congress acts in health care emergency

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Last week a young Florida child sank into a coma. Due to recent cutbacks, he
wasn’t eligible for the state CHIP program and with no health insurance and
precious little money available from her job at Wal-mart, his mother had skipped
on taking him to the ER until it was too late. With a sudden realization that
American health care was in crisis, late last Saturday night Congress passed a
bill completely changing the nation’s health care system. President Bush flew in
from yet another vacation to sign the new law, saying that the health care
system ought to preserve the culture of life. Evangelical religious groups
picketed Congress holding banners denouncing the impact of the lack of health
insurance and the high cost of health care for poor and middle class Americans.
Self-appointed moral values spokesmen denounced the nation’s courts for doing
nothing to improve the infant mortality rate, claiming that it was below that of
Cuba. Finally, all the cable news channels turned their networks over 24 hours a
day to covering the crisis. Even though there were bitterly opposing sides in the dispute and polls seemed
to show that most Americans didn’t agree with Congress or the President, the
politicians insisted that the moral imperatives made their action necessary. Sage editorials in the major newspapers explained that the Florida
tragedy had at least had the virtue of having families in the nation discuss
whether or not they wanted access to health insurance. Sadly the young boy died unaware that his fate had transformed a nation.

PHARMA/QUALITY: Can Cancer Care Get Better? by Greg Pawelski

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AP Biotechnology Writer, Paul Elias,  wrote an article this week that described how while the cost of cancer drugs have skyrocketed, the benefits are less apparent. It’s been more than 30 years since we declared a war on cancer and although there have been some real triumphs, and some great advances, the overall picture is not good. Tomorrow one of my closest friends is going into the local oncology center for the removal of what we all hope are some benign breast lumps. This post is dedicated to her, and to all those with cancer or at risk for cancer. Part of the issue is surely environmental, and we have much more to learn about what causes cancer and whether the toxins that we put into the planet are coming back to attack us.  Part of the issue, though, is how we approach cancer care.  THCB contributor Greg Pawelski has written before about the need for more chemosensitivity testing, and now writes on how we can use what we know to more effectively care for patients.


We have produced an entire generation of investigators in clinical oncology who believe that the only valid form of clinical research is to perform well-designed, prospective, randomized trials in which patients are randomized to receive one empiric drug combination versus another empiric drug combination. The problem is not with using the prospective, randomized trial as a research instrument. The problem comes from applying this time and resource-consuming instrument to address hypotheses of trivial importance (i.e. do most cancers prefer Pepsi or Coke?).

There are 60-80 different therapeutic drug regimens out there, any one or in combination can help cancer patients. The system is overloaded with drugs and underloaded with wisdom and expertise for using them. Government and academic clinical investigators have failed to support the individualization of chemotherapy through laboratory testing, in favor of attempts to identify "one size fits all treatments" through trial and error testing which has consumed tens of thousands of human lives. This entire effort has been a colossal failure and a colossal waste of human and financial resources.

One of the main problems in providing effective chemotherapy is the situation that every patient is unique. Tumors grow and spread in different ways and their response to treatment depends on these characteristics. The amount of chemotherapy that each patient can tolerate varies considerably from patient to patient. Therapeutic protocols currently in use are limited in their effectiveness because they are based on the results of clinical trials conducted on a general patient population, yet no two patients are alike. Chemosensitivity testing can help to improve the efficacy of cancer therapies on an individual patient basis.

Without the information provided by chemosensitivity testing, oncologists have the freedom to choose between multiple different drug regimens, all of which have approximately the same probability of working. Some of these regimens are highly profitable to oncologists. Other regimens are much less profitable. Pre-screen testing takes away a lot of this freedom to choose and narrows the selection to those drugs that have the highest probability to be successful but may have lower profitability for the oncologist. This cuts into the oncologist’s bottom line, though it benefits the patient.

The hallmark of the disease is heterogeneity, yet the powers that be insist on trying to homogenize it, rather than tailoring treatment to the individual nature of the disease. If we devoted 10% of the "one-size-fits-all" resources to developing and testing methods to individualize therapy, we’d have actually made some progress at lowering the costs of cancer drugs.

POLICY: Florida HIV mystery raises questions, by John Pluenneke

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A little
more than thirty days ago, a statistician at the Palm Beach Department of
Health accidentally sent out an internal email containing a list with the names
of more than 6,000 local residents with HIV and AIDS. Officials immediately shut down and scrubbed
the county network. Relieved supervisors
praised the rapid response of the county IT department, which
was able to eliminate all of the offending emails within 10 minutes. A lot of
people – including me – wondered how they could be so sure the problem had been
solved that easily. 

A month
later it looks as though somebody has gotten their hands on the list.  One by one,  mysterious letters have begun turning up at
the homes of people with HIV/AIDS across Florida. Not surprisingly, many of those people are
upset and quite understandably concerned that the  information could travel further.
The director of the Palm Beach County health department calls the
incident “medical terrorism” and says the letters remind her of the 2001 anthrax
attacks.  That may well be a little
strong.   

Like the
Kaiser Permanente story, the Palm Beach debacle has been
largely ignored by the media in the early going.  The issues the incident raises, however, are very
similar. How safe are patient  medical records, really?  What use are expensive network security
systems when human error can easily bypass their defenses?  If recent trends are any indication, these
cases are only the tip of the iceberg.  That
should be very worrying.

HIPAA was designed, at least in part, to prevent this kind of problem. The real
question at this point seems to be how effective is it really going to be at doing its job?  A little over a month from now, we’ll have a
very good idea.

County health officials in Palm Beach are arguing – in
the way officials often do – that last month’s email incident and this latest
problem are a complete coincidence.  That
boggles the imagination. Such coincidences just don’t happen. The
story itself
is made a little more interesting by the fact that the letters do not
appear to
be at all threatening. The problem is that they reveal information they
shouldn’t. Whoever is sending them appears to be motivated by a desire
to reach to the HIV/AIDS community and to simply have not thought
through his or her plan
very well.  This is one to follow.

INTERNATIONAL: The UK plans for a group practice future

Showing again that innovation isn’t dead in single-payer (and for that matter socialized) systems, the Brits are serious about creating the environment for pay for performance.  In an article called Super-surgery plan signals end for the family doctor, the Times reports that the British government is planning on coalescing its traditional onesy-twosey GP practices into bigger units.

While the article is full of fears about the end of Dr Finlay (the UK’s TV version of Marcus Welby), and also ridiculous claims that it’s being pushed because mass murdering GP Harold Shipman got away with his crimes because he had no partner looking over his shoulder, the real issue is that the Brits now believe that group practice in primary care will create better quality care.

They have already instituted a pay-for-performance model for primary care that rewards physician practices for hitting a number of process targets.  And over the last decade, they have spent the money and the necessary political capital to computerize practices.  Now they are going to force the GPs into the organizational form that has been shown in the states to create the environment for continuous care quality improvement, and of course have the shared resources to put in the systems that can monitor those processes.

Obviously there are lots of problems with the British system, and the connectivity both in terms of IT and in terms of communication between the primary care and the specialty care parts of the system is still an issue, and will be even when the huge IT project gets done — and that has its own problems.  But the fact remains that, despite all the knocks on the UK system, because it is government controlled it’s able to push the providers into a format that–according to the tenets of the best health services research–has been shown to produce the most effective medical care.

Of course, while there is a good game talked about that by Brailer and McClellan, they must be very envious of the relative power of their British equivalents.

PHARMA: Two quickies

The guy who was mentioned in a THCB post a while back about writing a tell-all book about being a slacker Viagra salesman has found that his new employer, Eli Lilly, didn’t approve, and fired him. I assume he’ll do well in his new role as a consultant on the next Michael Moore movie.

Meanwhile, John Mack apparently has his sights set on a bigger challenge than turning around Merck, (chortle, chortle).

PHARMA/POLICY: The NY Times misses the point on medicinal marijuana

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The New York Times has a very dumb article about medical marijuana called Medicinal Marijuana on Trial, which suggests that medical marijuana hasn’t been properly proved by clinical trials.

Well leaving aside that plenty of drugs have been approved by the FDA which have had less than full trials, there are three points not made in the article.

1st: Medical marijuana has been broadly favored by an Institute of Medicine report, and the Federal government has been sending it out for years to a very few patients.

2nd: There have been no major trials in the US, and precious few elsewhere, precisely because the DEA has stopped it for purely political reasons. So this article, which shows modest benefits from the few studies that have been done, but irrelevantly claims that this is outweighed by the impact on teenagers who smoke vast quantities of marijuana for no medical purpose. So we deny sick people medicine because of the actions of other people.  Well in that case, we shouldn’t allow Percocet for people coming out of surgery, because some people are heroin addicts.  Illogical rubbish and the NY Times should know better.

3rd: Most importantly, like Ginko, vitamins, fish oil and God knows how many other "cures", sick people take medical marijuana because they think it makes them feel better and healthier.  Making people feel better is the point of medical care. Rightly or wrongly people should be allowed to have what they believe to be medicine.  And despite some 75% of the country being in favor of allowing medical marijuana, it’s purely political grandstanding by extremist (and predominantly family values Christian) groups–being used as a front for the vast amount of money that the taxpayer has to pour into law enforcement–that has opposed sick people getting what they believe to be medicine.  Shameful.

HEALTH PLANS/POLICY: Another terrible patient story

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Paul Martin has a terrible story to tell of how his health plan let him down badly, and how the health care system is stopping him getting what seems necessary care. I of course only have his word for all this, but at THCB we from time to time try to remember that health care is about real patients with real problems.  And Paul certainly fits that description:

Here is a summary of some of the things I’ve experienced in dealing with our health care system over the past 12 years. After a major health insurer (“MHI”) bought out my school district’s insurance in 1998, they permanently terminated the one benefit which, at the time, appeared to be allowing me to make slow headway against MPS — the rare disease with which, it would later turn out, I had been misdiagnosed.
Every mediator, doctor, and lawyer who looked at my policy’s language agreed that the provision cited by MHI to justify its termination did not do so. The doctor trying to prescribe the treatment was one of the world’s leading authorities on MPS. My local specialist, a participating provider with MHI, wrote two forceful letters on my behalf, terming MHI’s decision against me "arbitrary."
MHI nevertheless terminated the benefit.

When, as a last resort, I started calling law firms, the first three I called couldn’t help because they were already being retained by MHI. Hundreds of letters and calls later, it became clear that no lawyer was going to take my case. Lawyers know the legal deck is stacked against them when it comes to defending patients against insurance corporations. Examples: boilerplate policy language that explicitly allows medical directors to overrule the treatment decisions of patients’ doctors. The deep pockets of insurance corporations that allow them not only to retain outside law firms, but their own legal staffs.

Two outcomes of MHI’s decision: 1) I became an "HMO refugee" – forced out of my job and out of my home state. Most of the school districts in my region were insured with MHI. How could I remain employed in a situation where I was covered for everything except the one treatment that all my doctors were recommending?  2) Watching my condition deteriorate, I took half my life’s savings and spent them on an experimental therapy with a good reputation that was less expensive than what my doctors wanted. The three weeks of therapy caused irreparable harm.

About a year ago, after experiencing increasing difficulty keeping outpatient appointments, I was permanently hurt on 2 out of 3 of my last doctor visits. My neuromuscular condition had become too fragile to allow safe transport by any means. Ever since, my family and I have been trying to arrange for physicians to come to my home. During this time, what began as a slightly impaired ability to take walks turned into the complete loss of that ability, then the loss of the ability to drive. By now I am only able to move about the house with a shuffling gait, and must spend increasing amounts of time in bed to alleviate pain.

As one example of the obstacles to home care that we have faced here in Delaware County PA (a suburb of Philadelphia): Delaware County Visiting Physicians does not accept Blue Cross/Blue Shield, only Medicare. I was granted Medicare as part of my social security benefit. However, social security does not allow the benefit to become active for two years.
Although my primary symptoms are soft tissue wasting and pain, I cannot receive pain medication. Federal law prohibits even immediate family members who are carrying the patient’s identification from obtaining pain scripts for the kind of serious (morphine-based) pain medication doctors were prescribing for me when I could still function as an outpatient. The patient either goes to the pain specialist’s office – at a minimum, every 30 days – or does without. (I worked as a school counselor for 23 years and have zero history of substance abuse, addiction to any drug, or even recreational drug use.)
The condition underlying my tissue wasting, severe osteoporosis, peripheral neuropathy, and a few enigmatic skin lesions "consistent with Sarcoidosis," remains unidentified.

Over the course of 12 years, I have proceeded from one specialist to another, usually at my own initiative. I have learned that a patient with an unusual and complex condition, arriving with the 50-page abridged version of his medical record, seldom receives more than the usual 10 minutes. There has never been a coordinated effort among specialists to reach a diagnosis or treatment recommendations. The minimal communication that has occurred among them has almost always been at my initiative.
In brief, a corporate dominated system of health care has no financial incentive to spend the extra time and resources to care adequately for patients falling outside the "best practices" box designed to meet the needs of the many. That’s where the money is.

PHARMA/PHYSICIANS: How to deal with the prostate conundrum

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Dame Edna was always making a running gag about her husband Norm’s enlarged prostate. But what should one do about it? Contributor Dr Krankheit (who as you might suspect is just playing one on television) has a solution:

The results of a recently completed project allow me to offer some tips in the interest of informed medical consumerism. It appears there is a major split among urologists in methods of treating benign prostatic hypertrophy
depending upon the specialist’s age.  Younger uros still possess the fantasy
image of themselves as surgeons and this disproportionately inclines them to
procedural/surgical fixes. In the case of BPH this means TURPs (transurethral
resectioning procedures) and TUNAs (transurethral needle ablations).  As with
older, neutered cats, older uros lose interest in these insertion procedures
because the callbacks from patients and evening/weekend rounds start to become a
drag.  Older uros instead prefer pharmaceutical remedies for BPH such as the
5-alpha reductase inhibitors (Proscar or Avodart).  So as an informed medical
consumer, if you find yourself getting up more than once a night to go to the
bathroom, you can choose to remedy the condition with someone putting a laser up
your wang and burning off part of your prostate or, alternatively, by taking
pills that may cause you to develop breasts (gynecomastia).  Select a
young buck urologist for the stick-and-burn option and one into his 50’s for the
D-cup treatment.

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